Clinical Trial

Novartis oral Fabhalta® (iptacopan) receives positive CHMP opinion for the treatment of adults living with C3 glomerulopathy (C3G)

If approved, Fabhalta® will be the only medicine indicated to selectively target the underlying cause of C3G1, an ultra-rare, progressive kidney…

10 months ago

Zevra Launches New Disease State Awareness Campaign, ‘Learn NPC, Read Between the Signs,’ to Drive Early Recognition and Treatment of Niemann-Pick Disease Type C

New website launched in honor of Rare Disease Day, Feb. 28, 2025 Awareness campaign includes educational resources and testing information…

10 months ago

Apellis Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results

Reported $781.4 million in full year 2024 revenues, representing 97% year-over-year growth SYFOVRE® (pegcetacoplan injection) full year 2024 net product…

10 months ago

Linvoseltamab Recommended for EU Approval by the CHMP to Treat Relapsed/Refractory Multiple Myeloma

TARRYTOWN, N.Y., Feb. 28, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Medicines Agency’s…

10 months ago

Akero Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Provides Business Update

-- Reported unprecedented, statistically significant reversal of compensated cirrhosis (F4) due to MASH after 96 weeks of treatment with efruxifermin…

10 months ago

Traws Pharma Regains Compliance with NASDAQ Equity Listing Rule

NEWTOWN, Pa., Feb. 28, 2025 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (Nasdaq: TRAW) (“Traws” or “Traws Pharma”), a clinical stage…

10 months ago

Opthea Reports Half Year Results and Business Updates

Cash and cash equivalents of $131.9M as of December 31, 2024 Cash runway extends through anticipated topline data readouts of…

10 months ago

ANI Pharmaceuticals Reports Record Fourth Quarter and Full-Year 2024 Financial Results and Raises 2025 Guidance

Generated record quarterly net revenues of $190.6 million, representing year-over-year growth of 44.8%Total Rare Disease quarterly net revenue of $87.0…

10 months ago

Avidity Biosciences Reports Fourth Quarter 2024 Financial Results and Recent Highlights

Building on success across its three clinical programs, Avidity is leading in rare neuromuscular diseases with a strong balance sheet…

10 months ago

Across Global Alliance Selects Wemedoo’s oomnia® as Central Clinical Trial Infrastructure

Across Global Alliance has partnered with Wemedoo AG to implement oomnia as its centralized clinical trial infrastructure, enhancing efficiency, compliance, and…

10 months ago